Posts by: Joana Carvalho, PhD

Joana Carvalho, PhD

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

October 14, 2020October 14, 2020

Interviews Reveal Women With Genetic Blood Disorders Share Uncertainties Regarding Diagnosis

News

Women with genetic blood disorders, including those who are carriers of hemophilia A and B, face multiple uncertainties surrounding their diagnosis, according to a recent interview-based study. Many women ... Read more

September 30, 2020October 2, 2020

CRISPR/Cas9 May Be Better Than Gene Therapy at Increasing FIX Activity

News

The gene-editing tool CRISPR/Cas9 may be superior to conventional gene therapy at increasing the activity of artificial factor IX (FIX), the blood-clotting protein missing in people with hemophilia B, ... Read more

September 18, 2020September 18, 2020

Roche Proposes Transfer Needle With Filter When Using Hemlibra

News

Reports of translucent particles of being detected in Hemlibra (emicizumab) in 2019 do not pose a safety risk or affect the therapy’s effectiveness and, according to Roche, health authorities ... Read more

August 21, 2020August 24, 2020

‘Huge Step Back’ for Hemophilia Gene Therapy, Expert Says of FDA Delay on Roctavian

News

The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others ... Read more

August 19, 2020August 21, 2020

FDA Delays Decision on Roctavian, Potential Hemophilia A Gene Therapy, for a Year or More

News

The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian, the potential first gene therapy for adults with hemophilia A. In ... Read more

July 24, 2020July 28, 2020

#ISTH2020 – Gene Therapy BAY 2599023 Showing Steady FVIII Activity, Safety in Trial

News

A single dose of the investigational gene therapy BAY 2599023 safely promotes a sustained production of factor VIII (FVIII), effectively preventing spontaneous bleeds in people with severe hemophilia A, a ... Read more

July 22, 2020July 27, 2020

#ISTH2020 – Eloctate Effective in Previously Untreated Hemophilia A Patients, Trial Shows

News

Treatment with Eloctate (efmoroctocog alfa) was well-tolerated and effective at preventing and treating bleeding episodes in children with severe hemophilia A who had never received any form of therapy ... Read more

July 20, 2020July 21, 2020

#ISTH2020 – BioMarin’s Gene Therapy Drives Sustained FVIII Production

News

A single infusion of valoctocogene roxaparvovec, BioMarin Pharmaceutical’s investigational gene therapy, can lead to the sustained production of factor VIII (FVIII) in people with hemophilia A, according to a ... Read more

July 17, 2020July 17, 2020

#ISTH2020 – FLT180a Gene Therapy Shows Promise for Hemophilia B Patients in Phase 1/2 Trial

News

A single dose of the investigational gene therapy FLT180a can lead to clinically meaningful, sustained increases in the activity of factor IX (FIX), effectively preventing bleeds and the need for ... Read more

July 15, 2020July 20, 2020

#ISTH2020 – Hemlibra Continues to Show Favorable Safety Profile, Early Phase 3b Trial Data Show

News

Note: This story was updated July 20, 2020, to note that Hemlibra binds to activated FIX and FX, rather than to FIX and FX. When given as preventive treatment ... Read more